To obtain a deeper understanding of cancer and speed the
development of new and improved cancer interventions, it
is essential to engage patients throughout the continuum
of research and care. Unfortunately, fewer than 5 percent
of adult cancer patients participate in a clinical trial (155).

Participation is even smaller among the elderly, women,
racial and ethnic minorities, and people living in rural
areas. To achieve outcomes relevant to all segments of
the population, it is essential to increase the number
and diversity of clinical research participants through a
combination of outreach, education, and policy changes
aimed at overcoming the barriers that prevent individuals
from participating in these studies.

However, advancing patient-centered cancer research
involves more than simply increasing the number and
diversity of patients participating in research. Research
policies should encourage the engagement of patients in the
conception, design, and dissemination of research in order
to address the questions that are most important to them,
their loved ones, and their caregivers; help researchers
to measure health outcomes from the perspectives of
patients; minimize the barriers to patient participation; and
ensure that research ;ndings are shared with the patient
communities that they are intended to bene;t.

Another way to advance patient-centered research is
to better integrate laboratory and clinical data, making
it possible for researchers to use data generated in the
clinic to answer scienti;c questions and helping health
care providers rapidly deliver care that is consistent with
the latest research ;ndings (see Figure 8, p. 33). To make
this vision a reality, it will be important to facilitate the
development of data infrastructure, standards, and policies
that enable the capture, aggregation, analysis, and utilization
of large volumes of high-quality clinical information while
protecting the rights and privacy of the patient community.

Maximize Opportunities in
Regulatory Science and Policy

Translating a deeper understanding of cancer biology into
a new medical product bene;tting cancer patients costs an
average of about $1 billion, and the process can take over
a decade (see Clinical Trials, p. 35). ;e growing ;eld of
regulatory science holds the key to improving e;ciencies in
this process.

Regulatory science is the study of developing new tools,
standards, and approaches to assess the safety, e;cacy,
quality, and performance of medical products (see sidebar
on Regulatory Science, p. 92) and is an integral part of FDA’s
review and decision-making processes. FDA’s regulatory
science initiatives are aimed at developing evidence-based
regulatory policies and expediting development of more
safe and e;ective medical products for cancer patients
everywhere.

;anks to the amazing progress made thus far against
cancer, as highlighted in this document, the FDA is
increasingly being asked to evaluate and regulate many
novel therapies and technologies, such as immunotherapies
and DNA sequencing. In many cases, the medical products
under review are so novel that the current means of
regulating them are inadequate. ;us, to continue or
accelerate the current pace of progress against cancer, it is
essential that advances in regulatory science parallel those
in basic, translational, and clinical science.

To advance regulatory science, regulators must have the
resources to support research that informs the regulatory
process, as well as a su;cient budget to recruit, develop, and
retain a highly quali;ed regulatory sta;. Likewise, enhanced
scienti;c exchange, cooperation, and collaboration
among stakeholders from academia, industry, advocacy,
and government are critical to advancing this ;eld.

Fostering meaningful exchange among these groups can
be accomplished by ensuring that regulators are permitted
to travel to national and international scienti;c meetings,
where they can be kept abreast of the latest developments

Jakafi
(Ruxolitinib),
a drug used to treat
myelofibrosis, was granted
FDA approval based on
novel endpoints, including
the impact of the drug on
symptoms reported by the
patients (patient-reported
outcomes).